Ethan Perlstein runs his lab like no other at UC San Francisco. In fact, he is not technically affiliated with UCSF – nor with any other university or organization. Instead, he is an independent researcher dedicated to discovering and developing targeted drugs for rare diseases (and some more common ones, as well).

Frustrated with the lack of attention given to such “orphan” diseases by academia and traditional funders, Perlstein went indie in 2012, setting up his group as a public benefit corporation called Perlara, backed by seed accelerator Y Combinator and how housed at the University of California biosciences incubator QB3. At Perlara, a diverse team with expertise in genetics, pharmacology, cell biology, data science, and automation share an enthusiasm for scientific discovery and a commitment to transparency and engagement.

Since orphan diseases typically involve rare mutations in individual genes, the Perlstein group uses CRISPR gene engineering to create several parallel animal models of these specific mutations, then rapidly screens through vast panels of chemicals for drug candidates that reverse the defects caused by those mutations.

Perlstein will speak to both his lab’s progress in tracking down treatments for orphan diseases, but also on his strategy for bypassing aspects of the academic research enterprise that he deems overly slow and conservative.